ATTR Amyloidosis ‘no longer the death sentence it once was’ after Patisiran approval in the South
The approval by the HSE in the South of a life-saving drug for Attr Amyloidosis means this ‘devastating disease in all its full-blown fury is no longer the death sentence it once was,’ campaigners have said.
The Attr Amyloidosis All Ireland Support Group received word through one of its supporters and advocates, Betty Holmes of Donegal Action for Cancer Care, that the Executive Management Team of HSE approved Patisiran in theSouth.
It is already available to patients in the North.
Campaigner Rosaline Callaghan told the Journal how the group was ‘thrilled and relieved at this decision.’
“It is a joyous good news story and our group has received congratulations and well-wishes from across the world.
“While we are delighted Patisiran is approved, it is dependent on a Managed Access Programme being implemented by HSE that would support the appropriate prescribing of this medicine.
“While the disease has a geographically specific origin in North West Donegal it has travelled throughout Ireland and across the world. At this stage, we understand around 30-35 patients with Hereditary Amyloidosisin in the 26 counties may be eligible for the treatment.
“I am one of 12 in NI receiving the treatment which has been available here since October 2019.
“We know that 50 million euro was allocated for new medicines in ROI in 2021 and funding for Patisiran will be from this allocation so we still have a lot of work to do to ensure that all patients who need it have speedy access to this ‘game-changing’, gene-silencing drug.
“We have been sustained in our efforts as a group over the past 14 months by generous and concerned people who have supported us and helped us raise awareness. We would like to thank Betty Holmes, Gerard Callaghan and the Burt Residents Group, Donegal County Council, Laura Glenn of the Derry Journal, Catherine McGinty of the Inish Times, Highland Radio, TG4, and other media who covered our stories.
“We have been supported by the Irish Heart Foundation, British Heart Foundation, UK Attr Amyloidosis Group, Rare Diseases Ireland, and the Northern Ireland Rare Diseases Partnership.
“We are grateful to Magee University, who worked with NIRDP, to have a module on Rare Diseases included in the Nurse Education curriculum to include a presentation on Hereditary Amyloidosis which was recently shared with 200 trainee nurses We thank the TDs and Ministers who advocated on our behalf. We thank the members of the public who took the time to speak with their local TDs and Ministers to seek their assistance on our behalf. I personally thank all of the group members who went public with their stories of Hereditary Amyloidosis in order to raise awareness. Our Group now numbers over 300 people and it has been humbling and emotional to speak with other people who believed they and their families were alone in dealing with this disease.”
Rosaline issued a special note of thanks to the admins of the group for their ‘tireless endeavours so far:’
James Green, Eibhlin Cunningham, Bernie Cannon Cunningham, Mary Halpin Byrne, Jennifer Doherty and Paddy Doherty.
She continued: “Our group remains committed to ensuring the best possible outcomes for Hereditary Amyloidosis patients which includes raising awareness of this disease and the early red flag symptoms. We shall press for speedy access to genetic testing and counselling, raise awareness with Health Care Providers so that a diagnosis is made as early as possible owing to the progressive nature of the disease which is ultimately fatal in the absence of treatment, and for access to Patisiran if prescribed, at the earliest opportunity.
“We are grateful to the Health Care Providers throughout the 32 counties who have been and remain committed to helping Amyloidosis patients. We are grateful for the National Amyloidosis Centre in London, which is the Centre for
Excellence for all Amyloidosis patients, and the team there are renowned world experts in the field. We are appreciative of the huge groundswell of support from our communities.
“If anyone would like to make contact with us they are most welcome to. This devastating disease in all its full-blown fury is no longer the death sentence it once was. Hereditary Amyloidosis patients now have hope where previously there was none.
“We will do everything we can to help support patients and their families and can be contacted through
our Support Group
our Awareness Page